A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates

Adeno-associated virus (AAV) has provided the gene therapy field with the most powerful in vivo gene delivery vector to realize safe, efficacious, and sustainable therapeutic gene expression. Because many clinically relevant properties of AAV-based vectors are governed by the capsid, much research e...

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Detalles Bibliográficos
Autores principales: Wang, Dan, Li, Shaoyong, Gessler, Dominic J., Xie, Jun, Zhong, Li, Li, Jia, Tran, Karen, Van Vliet, Kim, Ren, Lingzhi, Su, Qin, He, Ran, Goetzmann, Jason E., Flotte, Terence R., Agbandje-McKenna, Mavis, Gao, Guangping
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948233/
https://www.ncbi.nlm.nih.gov/pubmed/29766031
http://dx.doi.org/10.1016/j.omtm.2018.03.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948233/
https://www.ncbi.nlm.nih.gov/pubmed/29766031
http://dx.doi.org/10.1016/j.omtm.2018.03.004

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